Vertex Gains FDA Approval for ALYFTREK™, a Daily CFTR Modulator for Cystic Fibrosis

27 December 2024
Vertex Pharmaceuticals has announced the FDA approval of ALYFTREK™, a once-daily cystic fibrosis treatment designed for patients aged six and above, who have at least one F508del mutation in their CFTR gene or other mutations responsive to ALYFTREK. This approval marks Vertex’s fifth CFTR modulator to receive FDA approval, showcasing the company’s ongoing commitment to addressing cystic fibrosis's root causes and enhancing treatment for more individuals. According to Vertex's CEO, Reshma Kewalramani, ALYFTREK signifies a crucial step in their mission to improve CFTR function in patients through innovative treatment options.

The approval follows an extensive Phase 3 clinical program involving over 1,000 participants across more than 20 countries. The studies demonstrated ALYFTREK's non-inferiority to TRIKAFTA® in terms of absolute changes in ppFEV1, while achieving a significant reduction in sweat chloride levels compared to TRIKAFTA. The trials included participants aged 12 and older, as well as children aged 6-11, with ALYFTREK meeting primary endpoints for safety and efficacy in both age groups. Furthermore, the treatment was generally well-tolerated across the studies.

Dr. Claire Keating from Columbia University, who was involved in the clinical trials, sees ALYFTREK as a potential advancement in cystic fibrosis care, offering improved treatment with its significant impact on sweat chloride levels. ALYFTREK is notable for being the first once-daily CFTR modulator, addressing a significant need for more convenient dosing among cystic fibrosis patients. This feature could benefit patients who must take their medication with fat-containing food. With this approval, approximately 150 individuals in the U.S. with specific mutations can access a CFTR modulator therapy for the first time.

Beyond the U.S., ALYFTREK is under regulatory review in several regions, including the European Union, United Kingdom, Canada, Switzerland, Australia, and New Zealand, indicating its potential global reach.

Cystic fibrosis, the genetic disease ALYFTREK aims to treat, affects over 92,000 people worldwide. This condition impacts multiple organs, including the lungs and pancreas, due to mutations in the CFTR gene. The disease is typically progressive and life-shortening, with most patients dying in their 30s without intervention. The introduction of ALYFTREK is a promising development in extending the lives of these patients by improving CFTR protein function, ultimately addressing the disease's underlying causes.

Vertex Pharmaceuticals continues to innovate in the field of cystic fibrosis and other severe genetic diseases, reinforcing its position as a leader in biotechnology. The company’s dedication to scientific advancement is exemplified not only in their cystic fibrosis treatments but also in their exploration of therapies for other serious conditions, such as sickle cell disease and various kidney diseases.

Overall, ALYFTREK represents a significant advancement in the treatment of cystic fibrosis, offering a once-daily regimen that could simplify care for patients and expanding therapeutic options for those with specific CFTR mutations. This approval reinforces Vertex's ongoing efforts to transform the landscape of cystic fibrosis treatment, improving the quality of life for affected individuals globally.

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